Myotonic Dystrophy Type 1 (DM1)
NVSNovartisCatalyst overview
Research summary
This catalyst tracks a Phase 3 data readout for Delpacibart Etedesiran (Del-desiran) (AOC 1001) in Myotonic Dystrophy Type 1 (DM1). The expected timing is based on the latest available company guidance and may change.
Asset from Avidity acquisition. MARINA, MARINA-OLE, HARBOR trials. 10/3/24- FDA lifted clinical hold. 7/28/25- Completed enrollment in HARBOR trial. 11/10/25- MARINA-OLE publication in Q4'25. 54wk topline data in H2 2026. 02/26/26:- MDA 2026 posters on DM1 patient journey and del-desiran MARINA-OLE treatment-experience interviews on March 9, 2026.
Verification
Confirm timing and program details against the company announcement or filing.